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Safety, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged >12 Years) With Fabry Disease

NCT04049760 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2026-02-05

Study results available
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Summary

This is a long-term, Open-label Study to Evaluate the Safety, Pharmacodynamics, and Efficacy of Migalastat in Subjects \> 12 Years of Age With Fabry Disease and Amenable GLA Variants

Conditions

Interventions

DRUG

migalastat HCl 150 mg

migalastat HCl 150 mg capsule

Sponsors & Collaborators

  • Amicus Therapeutics

    lead INDUSTRY

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2019-10-11
Primary Completion
2024-11-29
Completion
2024-11-29
FDA Drug
Yes

Countries

  • United States
  • United Kingdom

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04049760 on ClinicalTrials.gov